Date: 2016-09-01
Type of information: Granting of the orphan status in the US
Product name: SB-FIX - adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and human factor 9 gene (SB-FIX)
Compound: adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and human factor 9 gene (SB-FIX)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Action mechanism:
- genome editing product/gene therapy. SB-FIX is designed as a single treatment strategy intended to provide stable, continuous production of Factor IX clotting protein (FIX) for the lifetime of the patient. Sangamo's ZFN-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic "safe-harbor site," that can be edited with zinc finger nucleases to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein product. The ability to permanently integrate the therapeutic gene in a highly specific targeted fashion significantly differentiates Sangamo's in vivo genome editing approach from conventional AAV cDNA gene therapy approaches, which are non-integrating, and may "wash out" of the liver as cells divide and turn over.
Company: Sangamo Therapeutics (USA - CA) previously known as Sangamo Biosciences
Disease: hemophilia B
Latest news:
- • On September 1, 2016, the FDA has granted orphan drug designation for adeno-associated virus serotype 2/6 (rAAV2/6) vectors encoding zinc finger nucleases (ZFNs) and human factor 9 gene (SB-FIX) for the treatment of hemophilia B. Sangamo expects to initiate a Phase 1/2 clinical study (SB-FIX-1501) in adult subjects with the disease in 2016.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2016-09-01
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
